Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Clin |
RCV001290701 | SCV001478838 | likely pathogenic | Hereditary thrombocytopenia and hematologic cancer predisposition syndrome | 2021-01-12 | reviewed by expert panel | curation | This late frameshift insertion is not expected to result in nonsense mediated mRNA decay, but will alter the transactivation domain/inhibitory domain/VWRPY motif and elongate the protein [PVS1_strong]. It is absent from gnomAD (v2 and v3) [PM2]. The variant has only been reported in one proband with AML, however the germline origin is unclear (SCV000807758.1). It is of note that other C-terminal frameshift variants have been reported with some showing loss of transactivation ability and a dominant-negative effect (PMID: 14615365, 19808697, 25840971). In summary, the clinical significance of this variant is likely pathogenic. ACMG/AMP criteria applied, as specified by the ClinGen Myeloid Malignancy Variant Curation Expert Panel for RUNX1: PVS1_strong and PM2. |
Prevention |
RCV000680387 | SCV000807758 | likely pathogenic | not provided | 2018-07-12 | criteria provided, single submitter | clinical testing |